Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study

Background: Pyruvate kinase (PK) deficiency is a rare hereditary anemia caused by mutations in the PKLR gene encoding the red blood cell (RBC) PK enzyme (PKR). Defects in PKR lead to chronic hemolytic anemia, which is associated with serious complications, regardless of transfusion status.